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Taking people in United StatesPhase 1

Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)

Key facts

Where
Houston, United StatesSee the site
Phase
Phase 1

Where in United States

  • The University of Texas Health Science Center at HoustonHouston, United StatesTaking people

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Trial details

Who runs it
The University of Texas Health Science Center, Houston
Trial code
NCT06280482
Record updated
12 September 2025
Original description
The purpose of this study is to determine whether SMDS patients treated with NR at the proposed dose exhibit decreased glucose uptake in the aorta, to determine if NR treatment results in measurable changes of blood NAD+ and NR levels, to determine if aortic measurements are stable after treatment with NR and to evaluate the safety and tolerability of NR in SMDS patients.Read full original descriptionShow less

The purpose of this study is to determine whether SMDS patients treated with NR at the proposed dose exhibit decreased glucose uptake in the aorta, to determine if NR treatment results in measurable changes of blood NAD+ and NR levels, to determine if aortic measurements are stable after treatment with NR and to evaluate the safety and tolerability of NR in SMDS patients.

Inclusion: Individuals diagnosed with SMDS with confirmed ACTA2 mutation disrupting arginine 179; Parental/guardian permission (informed consent) and, if appropriate, child assent.

Exclusion: Individuals who have undergone surgery to replace aneurysmal or dissected ascending and root aortic tissue with a graft; Prior consumption of niacin or nicotinamide riboside supplement within the prior eight weeks; Concurrent participation in another intervention trial; Additional medical conditions that impair the patient's ability to participate in the study; Known allergy or sensitivity to niacin or nicotinamide riboside; Failure to provide informed consent.

ClinicalTrials.gov NCT06280482